ISSN 1662-4009 (online)

ey0015.12-13 | New treatments | ESPEYB15

12.13 Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children with Heterozygous Familial Hypercholesterolemia: The CHARON Study (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label)

M Braamskamp , G Langslet , BW McCrindle , D Cassiman , GA Francis , C Gagne , D Gaudet , KM Morrison , A Wiegman , T Turner , E Miller , DM Kusters , JS Raichlen , PD Martin , EA Stein , JJP Kastelein , BA Hutten

To read the full abstract: Circulation 2017;136:359-366The National Heart, Lung and Blood Institute Panel on integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents recommended a universal screening approach that would include one-time testing of all children aged 9-11 years for dyslipidemia. This recommendation has raised controversy regarding t...

ey0015.14-17 | Who could have predicted it? | ESPEYB15

14.17 Exenatide once weekly versus placebo in Parkinson’s disease: a randomised, double-blind, placebo-controlled trial

D Athauda , K Maclagan , SS Skene , M Bajwa-Joseph , D Letchford , K Chowdhury , S Hibbert , N Budnik , L Zampedri , J Dickson , Y Li , I Aviles-Olmos , TT Warner , P Limousin , AJ Lees , NH Greig , S Tebbs , T Foltynie

To read the full abstract: Lancet 2017;390:1664-1675Whoever could have imagined that a compound discovered as being secreted by enteroendocrine cells in the gut could have a role in treating Parkinson’s disease? GLP-1 does indeed have diverse actions on several peripheral organs (including tongue, stomach, adipose tissues, muscle, pancreas and liver) and on the brainstem to regulate...

ey0020.3-14 | Advances in Growth, Bone Biology, and Mineral Metabolism | ESPEYB20

3.14. SIRT2 regulates extracellular vesicle-mediated liver-bone communication

L Lin , Z Guo , E He , X Long , D Wang , Y Zhang , W Guo , Q Wei , W He , W Wu , J Li , L Wo , D Hong , J Zheng , M He , Q Zhao

In Brief: These authors studied liver-specific SIRT2 knockout mice to examine how loss of hepatocyte SIRT2 (Sirtuin 2) prevents bone loss in aged mice. Hepatocyte SIRT2 deficiency led to upregulation of Leucine rich α2 glycoprotein (LRG1) in hepatocyte-derived small extracellular vesicles (sEVs) which inhibited osteoclastogenesis in bone marrow.Commentary: Liver-bone communication has been implicated in bone homeostasis. P...

ey0020.7-2 | Gonadal Function and Fertility Issues in Childhood Cancer Survivors | ESPEYB20

7.2. Towards an individualized management of pubertal induction in girls with hypogonadism: insight into the best replacement outcomes from a large multicentre registry

G Rodari , S Federici , T Todisco , G Ubertini , A Cattoni , M Pagano , F Giacchetti , E Profka , V Citterio , D Messetti , V Collini , D Soranna , E Carbone , M Arosio , G Mantovani , L Persani , M Cappa , M Bonomi , C Giavoli

Brief summary: This longitudinal observational multicentre retrospective study, collected data on 95 young prepubertal or early pubertal girls (age >10.9 years, Tanner stage ≤2) with premature ovarian failure (POI) or hypogonadotropic hypogonadism (HH). Their hypogonadism was due to different causes and was treated with transdermal 17β-oestradiol, with a follow-up of at least 1 year. The study aimed to identify the most physiological and effective therapeutic sc...

ey0020.8-15 | New Hopes | ESPEYB20

8.15. Exocrine pancreas regeneration modifies original pancreas to alleviate diabetes in mouse models

X Kou , J Liu , D Wang , M Yu , C Li , L Lu , C Chen , D Liu , W Yu , T Yu , Y Liu , X Mao , A Naji , T Cai , L Sun , S Shi

Brief summary: In this experimental study, pancreas-derived mesenchymal stem cells (PMSCs) were implanted into the kidney capsule of mice with streptozotocin (STZ)-induced diabetes. PMSCs led to increased levels of IL-6 in T-helper 1 and T-helper 17 cells, which transiently activated tumor necrosis factor-alpha (TNF-α) and interferon-gamma (IFN-γ), which in turn decreased levels of interleukin-17. This was associated with exocrine pancreas regeneration and rescue of ...

ey0018.2-10 | Neonatal diabetes mellitus | ESPEYB18

2.10. NKX2-2 mutation causes congenital diabetes and infantile obesity with paradoxical glucose-induced ghrelin secretion.

A Auerbach , A Cohen , N Ofek Shlomai , A Weinberg-Shukron , S Gulsuner , MC King , R Hemi , E Levy-Lahad , A Abulibdeh , D Zangen

J Clin Endocrinol Metab. 2020 Nov 1;105(11):dgaa563. doi: 10.1210/clinem/dgaa563. PMID: 32818257.This manuscript describes an unusual case of a baby born with a homozygous Nkx2.2 mutation who developed severe neonatal diabetes mellitus and then on follow up went onto develop severe obesity characterized by marked hyperphagia.Nkx2.2 is an important transcription factor...

ey0018.3-2 | Thyroid hormone action | ESPEYB18

3.2. A coregulator shift, rather than the canonical switch, underlies thyroid hormone action in the liver

Y Shabtai , NK Nagaraj , K Batmanov , YW Cho , Y Guan , C Jiang , J Remsberg , D Forrest , MA Lazar

Genes Dev. 2021;35(5–6):367–378. doi: 10.1101/gad.345686.120.The longstanding concept of thyroid hormone (TH) action is summarized as the canonical switch model. This study adds important aspects of TH action to our current understanding, modifying this longstanding switch model to a “shift” model.According to the current concept, TH dependent gene...

ey0018.5-5 | Advances in clinical practice | ESPEYB18

5.5. Growth curves for children with X-linked hypophosphatemia

Mao Meng , Carpenter Thomas O , Whyte Michael P , Skrinar Alison , Chen Chao-Yin , Martin Javier San , Rogol Alan D

J Clin Endocrinol Metab. 2020 Oct 1;105(10):3243–3249 Abstract: https://pubmed.ncbi.nlm.nih.gov/32721016/In brief: X-linked hypophosphatemia (XLH) is the most common hereditary form of rickets and osteomalacia. The study used retrospective, pre-burosumab growth data from four different studies and constructed growth charts that demonstrate that the growth rate of children with...

ey0018.10-9 | (1) | ESPEYB18

10.9. Serum 25-hydroxyvitamin D concentration in childhood and risk of islet autoimmunity and type 1 diabetes: the TRIGR nested case-control ancillary study

ME Miettinen , S Niinisto , I Erlund , D Cuthbertson , AM Nucci , J Honkanen , O Vaarala , H Hyoty , JP Krischer , M Knip

Diabetologia. 2020;63(4):780–787. doi: 10.1007/s00125-019-05077-4.This multicenter multinational study, in children with high genetic risk of T1D, identified an association between lower serum 25-hydroxyvitamin D (25OHD) concentrations and subsequent appearance of islet autoimmunity or T1D.The Trial to Reduce IDDM in the Genetically at Risk (TRIGR) ancillary ...

ey0019.3-4 | Thyroid development | ESPEYB19

3.4. Transcriptomic signature of human embryonic thyroid reveals transition from differentiation to functional maturation

G Dom , P Dmitriev , MA Lambot , Vliet G Van , D Glinoer , F Libert , A Lefort , JE Dumont , C Maenhaut

Front Cell Dev Biol. 2021 Jun 11;9:669354. doi: 10.3389/fcell.2021.669354. eCollection 2021. PMID: 34249923Brief Summary: This in vitro study analyzed the transcriptome of the developing thyroid gland in human embryonic thyroids compared to non-thyroidal human embryonic tissues and adult thyroid and adult non-thyroidal tissues. They identified four differently regulated sets of gen...